Testing a New Oncology Drug Development Paradigm — Section 3

Together, DCTD and CCR investigators will utilize the recently announced Food and Drug Administration exploratory IND guidance to facilitate testing of targeted therapies in patients earlier in the drug development process so that informed decisions to proceed with or stop development can be made before expensive bulk drug formulation occurs. These studies will also take advantage of new advances in molecular imaging, which can help detect whether an agent being tested is reaching its target and having the desired effect.

A unique aspect of the program is that extramural drug developers, for the first time, will be offered opportunities to utilize CCR resources for clinical trials support. Candidate compounds for exploratory IND studies may come from intramural, extramural, academic NCI-funded, or industry laboratories. Consideration will be given to novel small molecules, antibodies, or peptide therapeutics.

Proposed exploratory INDs may start by obtaining PK data suggesting that appropriate drug levels in plasma and tumor can be achieved. Next, PD studies exploring how the agent affects its proposed target in vivo would be appropriate. Exploratory IND studies embody the ideal drug development scenario required to conduct a limited, single-dose PK/PD dose-escalation study in humans. In such trials, which are sometimes referred to as Phase 0 studies, researchers perform real-time PK/PD studies to guide dose escalation instead of escalation to maximum tolerated dose as is now the norm in phase I trials. This approach is essential for patient safety in early human clinical trials.